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Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Pompe Disease (Late-onset) Clinical Trial

Official title:
A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease

Clinical Trial Summary

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06391736
Study type Interventional
Source GeneCradle Inc
Contact GeneCradle, Inc China
Phone 86-13501380583
Email ind@bj-genecradle.com
Status Recruiting
Phase Phase 1/Phase 2
Start date April 19, 2024
Completion date December 2026
View Details
See also
  Status Clinical Trial Phase
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Recruiting NCT04174105 - Gene Transfer Study in Patients With Late Onset Pompe Disease Phase 1/Phase 2
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT03285126 - Pompe Gene Therapy- Screening for Eligibility
Recruiting NCT05083806 - MSOT in Pompe Disease N/A
Recruiting NCT05272969 - Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Active, not recruiting NCT03694561 - Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Completed NCT00455195 - Late-Onset Treatment Study Extension Protocol Phase 4
Completed NCT00731081 - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® N/A
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT01288027 - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Phase 4
Recruiting NCT00567073 - Pompe Pregnancy Sub-Registry
Completed NCT03893240 - Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease N/A
Withdrawn NCT04094948 - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Phase 2
Completed NCT00250939 - A Study of rhGAA in Patients With Late-Onset Pompe Disease Phase 2
Recruiting NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Phase 3
Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Not yet recruiting NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) Early Phase 1
Completed NCT00268944 - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support Phase 3